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NIH Pediatrics Research Report FY2019

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NIH Pediatrics Research Report FY2019 DEPARTMENT OF HEALTH AND HUMAN SERVICES National Institutes of Health Report to Congress: Pediatric Research in Fiscal Year 2019 September 2020 1 DEPARTMENT OF HEALTH AND HUMAN SERVICES National Institutes of Health Report to Congress: Pediatric Research in Fiscal Year 2019 TABLE OF CONTENTS Table of Contents ....................................................................................................................... i PEDIATRIC RESEARCH AT THE NATIONAL INSTITUTES OF HEALTH................................... 1 NEW IN FY 2019 ...................................................................................................................... 1 THE PEDIATRIC RESEARCH INITIATIVE................................................................................ 2 SELECTED SCIENTIFIC RESEARCH ADVANCES IN PEDIATRICS ........................................... 3 Pregnancy and Newborn Health 3 Child Development 6 Social and Environmental Influences 7 Nutrition and Obesity 10 Diabetes 12 Childhood Diseases 13 Immunity and Allergies 17 Rare Pediatric Diseases 19 Pediatric Cancer 19 Bone and Muscle Health 23 Oral Health, Speech, Hearing, and Vision 25 Intellectual and Developmental Disabilities, Neurological Disorders, and Mental Health 26 Childhood Injuries, Maltreatment, and Violence 31 Substance Use and Misuse 32 Pediatric Critical Care and Emergency Care 35 Clinical Care, Outreach, and Services 37 Pediatric Pharmacology 38 Technology and Tools 39 Global Pediatric Health 40 SELECTED NEW AND EXPANDED RESEARCH EFFORTS FOR FY 2019 IN PEDIATRICS ........42 Selected New Pediatric Research Efforts 42 Selected Expanded Pediatric Research Efforts 53 SELECTED MAJOR ONGOING NIH PROGRAMS IN PEDIATRIC RESEARCH...........................57 Pregnancy and Newborn Health 57 Child Development 60 Social and Environmental Influences 62 i Nutrition and Obesity 63 Diabetes 64 Childhood Diseases 64 Immunity and Allergies 67 Rare Pediatric Diseases 68 Pediatric Cancer 69 Bone and Muscle Health 73 Oral Health, Speech, Hearing, and Vision 73 Intellectual and Developmental Disabilities, Neurological Disorders, and Mental Health 74 Childhood Injuries, Maltreatment, and Violence 76 Substance Misuse 77 Pediatric Critical Care and Emergency Care 79 Clinical Care, Outreach, and Services 80 Pediatric Pharmacology 81 Technology and Tools 81 Global Pediatric Health 82 Other Cross-Cutting Areas 83 Research, Training, Career Development, and Loan Repayment Programs 84 ADDITIONAL PEDIATRIC COLLABORATIONS ......................................................................86 APPENDIX .............................................................................................................................88 Table 1: All NIH Pediatric Research, FY 2019 89 Table 2: NIH Funding Opportunity Announcements That Solicited Applications for Pediatric Research, Published in FY 2019 90 Table 3: Acronyms for NIH Institutes, Centers, and Offices and Other U.S. Federal Agencies Referred to in This Report 101 ii PEDIATRIC RESEARCH AT THE NATIONAL INSTITUTES OF HEALTH Research advances supported by the National Institutes of Health (NIH) have transformed the diagnosis, treatment, and prevention of disease. Children have benefitted greatly from revolutionary progress in biomedical research. Taking folic acid before and during pregnancy prevents most neural tube defects. Survival rates for preterm infants have increased substantially. The genetic causes of several disorders have been identified, such as Fragile X syndrome and Rett syndrome. Several conditions that once caused intellectual disability, like congenital hypothyroidism or phenylketonuria (PKU), are no longer major threats. Scientists’ understanding of how children grow and develop has grown immensely and informed early intervention efforts that have dramatically improved the lives of millions of children worldwide. Pediatric research continues to be an NIH priority. NIH’s strong basic and clinical research portfolio provides the foundation for pediatric research in a variety of scientific areas, including neurodevelopment, cardiology, cancer, pharmacology, and behavioral and social sciences. In fiscal year (FY) 2019, NIH funded research grants and projects directed specifically at pediatric research for a total of $4,922,180,825, as detailed in Table 1 in the Appendix. The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) funds the largest portion of pediatric research among the NIH institutes, centers, and offices (ICOs), taking a leadership role in many pediatric research efforts that involve trans-NIH collaborations. For example, NICHD leads the Trans-NIH Pediatric Research Consortium (N-PeRC), which was established in June 2018 to coordinate pediatric research programs, best practices, and training opportunities across all NIH ICOs. All the ICOs support pediatric research; NICHD alone accounts for only 18 percent of the total NIH support for pediatric research. This reflects the breadth of the research portfolio at NIH dedicated to improving the health of children everywhere. NEW IN FY 2019 In FY 2019, several new technologies for pediatric populations emerged from NIH-supported research. For example, NICHD-supported researchers applied artificial intelligence techniques to diagnose newborn screening results more rapidly, providing timely information to inform treatment decisions. A new handheld imaging tool, supported by National Eye Institute (NEI) researchers, improved diagnostics for infants with vision impairment. A wearable tool supported by the National Institute of Biomedical Imaging and Bioengineering (NIBIB) and the NICHD, coupled with standard therapy, increased socialization skills in children with autism spectrum disorder more than children who received only the standard therapy. Several interventions, initially supported by NIH funding, were approved by the Food and Drug Administration (FDA). For example, the first gene therapy approved for treating spinal muscular atrophy (SMA) was originally supported by the National Institute of Neurological Disorders and Stroke (NINDS). In another example, based on the promising results of a phase II multicenter trial, the Food and Drug Administration (FDA) awarded selumetinib with a breakthrough therapy designation.1 This designation allows the FDA to speed up the review process and possible approval of this drug. Selumetinib is being used to treat symptomatic and/or progressive neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN) in children age 3 and older. In NF1, a signaling pathway that causes tumor growth is turned on. The researchers found that selumetinib blocks a protein in this signaling pathway and reduces the size of tumors. The researchers also found that NF1 patients who were treated with selumetinib experienced improvements in clinical outcomes, such as less pain, more strength, 1 On April 10, 2020, the FDA approved Koselugo (selumetinib) for the treatment of pediatric patients with NF1. Koselugo is the first drug approved by the FDA for the treatment of NF1. 1 and better quality of life. After a year of treatment, most patients (or their parents) reported improved pain scores, strength, and range of motion. NIH also launched several initiatives that will provide insight into child development and childhood disorders. The NIH Baby Toolbox (NBT) is a comprehensive developmental assessment tool for infants and toddlers ages 1 month to 42 months that allows researchers and clinicians to evaluate brain development in infants and toddlers using a computer tablet. Supported by the trans-NIH Helping to End Addiction Long-term℠ (HEAL) initiative, the HEALthy Brain and Child Development Study will follow a large population of children from the prenatal period to age 10 to examine the impact of early exposure to opioids, other substances, and social stressors on brain development in children. In 2019, the Trans- NIH Fragile X Coordinating Committee published the NIH Strategic Plan for Research on FMR1- Associated Conditions, recognizing gaps and opportunities in studying a gene that plays an important role in the development and function of the brain and nervous system. Pediatric research at NIH has made great progress in characterizing typical development, diagnosing potential disorders, and designing interventions to improve overall health. THE PEDIATRIC RESEARCH INITIATIVE In the Public Health Service Act (the Act), Congress directed the Secretary of Health and Human Services (HHS) to establish a Pediatric Research Initiative (PRI) in the NIH Office of the Director (OD). The Act also directed the OD to “… annually report to Congress and the public on the extent of the total funds obligated to conduct or support pediatric research across the National Institutes of Health, including the specific support and research awards allocated through the Initiative.” – Section 409D(c)(3), Public Health Service Act In response to this directive, NIH has prepared the following report for FY 2019. The overall purpose of the PRI is to “conduct and support research that is directly related to diseases, disorders, and other conditions in children” (Section 409D(a), Public Health Service Act). More specifically, the purpose of the PRI is (1) “to increase support for pediatric biomedical research within the National Institutes of Health to realize the expanding opportunities for advancement in scientific investigations and care for children; (2) to enhance collaborative efforts among the Institutes to conduct
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